Derivation of New Human Embryonic Cell Lines in Full Compliance with Current FDA Regulatory Guidance

The research we propose develops improved safety procedures for the establishment of new human embryonic stem cell lines to treat disease. Although it addresses critical safety concerns necessary to move these cells into the realm of human disease treatment, this work could not be funded currently by the federal government.
Human embryonic stem cells are used by scientists to study basic questions about biology and how cells and tissues grow. There is also much hope that these same cells will eventually be used also to treat patients. To achieve safety when it becomes possible to use them to treat patients, the collection and handling of cells must be very different from the way the same cells would be handled if they were to be used only for laboratory study. The work we propose is intended to make a complete assessment of safety practices in the way embryonic stem cells are presently handled from the time they are donated to the time they are cultured and frozen for future use. By making this study, we will be able to identify ways to improve safety. We will do this by observing the Food and Drug Administration's regulations for safety in using cells that are intended for use in treating human disease. At the present time, safety procedures are not well developed and it is crucial that they be greatly improved before embryonic stem cells can be used to treat human disease. We believe that these studies will both make stem cells safer to use for people and make such uses for people available more rapidly.

Statement of Benefit to California:

The research we propose is concerned with developing improved safety procedures for the establishment of new human embryonic stem cell lines to treat human disease. Although it addresses critical safety concerns necessary to move these cells into the realm of human disease treatment, this work could not be funded currently by the federal government.
When the voters of California approved Proposition 71, they anticipated that research done under its funding would lead to treatment of human diseases and to economic benefits to California's citizenry, although it is understood that much more basic research is also needed before this is to happen. Our work will pave the way for treatment of any human disease using embryonic stem cells by making a detailed and comprehensive study of safety procedures needed for the establishment of new embryonic stem cell lines. The currently approved federal cell lines have both biologic and safety limitations. Therefore, it is critical that new stem cell lines are established in full compliance with existing FDA safety regulations. A method to accomplish this has not yet been devised and we propose to address this barrier to safety and clinical use of human stem cells to treat diseases. This work will benefit California directly because the state will become known as the first to comprehensively address the safety issue. In addressing this issue, the clinical use of stem cells anticipated by Cailfornia's voters will be greatly facilitated while simultaneously being made more safe. In this environment, it will also become more likely that additional biotechnology companies, whose business models will be based on cellular therapy with stem cells, will be established and thrive in the state. these companies will pay taxes and provide employment.